Key CDER Priorities Need Funding Too

Until we have the budget-in-detail for FDA (also known as the CJ or Congressional Justification), we have relied on Commissioner Gottlieb’s statement on “new FDA funding to promote innovation and broaden patient access through competition.” At her meeting with the Alliance, Dr. Woodcock reviewed the programs in the budget request that would be led by CDER and which represent the Center’s five priorities for investment.Outsourcing Facilities.  Outsourcing facilities became a new category of drug manufacturer under the Drug Quality and Security Act of 2013 (DQSA). Dr. Woodcock explained that adequate funding for DQSA implementation is a priority for CDER leadership because outsourcing facilities provide important medicines not otherwise commercially available. However, the requirements of the Act are rigorous and the industry needs help to meet their statutory obligations, protect consumers, and decrease drug product shortages.Knowledge Management Systems. Dr. Woodcock explained that an ongoing challenge in the Office of New Drug Products (ONDP) is the inability to capture, track, and research prior decisions impacting drug review and development. Readily accessible information within the new system would increase efficiency, consistency, and predictability in regulatory decision-making. She pointed, particularly, to the opportunity to provide more uniform advice to companies in early-stage development, reduction of inter-review division variability, and the likelihood this will reduce the number of one-off decisions and the number of products requiring multiple review cycles.Real-World Evidence (RWE). The appropriate uses of RWE are still in the early stages, but have the potential to improve patient health and support pre-market evaluation and post-market safety. Much like it has done with biosimilars, CDER is looking to work with sponsors on applications of RWE and case studies and leave more formal guidance until the agency has more experience with RWE. Dr. Woodcock identified new indications/uses for already approved drugs and post-approval reporting of patient experiences as possible candidates for use of RWE. To take advantage of this opportunity, CDER needs funding to develop data and analytical tools to collect and evaluate RWE.Advanced Manufacturing Initiatives. There is enormous opportunity for transformation of the manufacturing process for drug (and device) products, with the potential of improved efficiencies, lower costs, and the movement of more manufacturing back to the US. One major item is the possibility of changing over from batch-process to continuous manufacturing, but there are multiple opportunities for innovation in manufacturing. However, current good manufacturing practices (GMP) regulations have the effect of suppressing innovation and greatly add to the risks for a company trying to manufacture based on alternative approaches. FDA wants to get ahead of this -- to work with companies on alternatives and create guidances and regulations that enable manufacturing innovations.Rare Diseases. Despite the obvious success in development of therapies to treat rare diseases, there are a number of methodological challenges that need significant additional work. Dr. Woodcock pointed to the challenges of small clinical trials and the potential to simplify drug development for rare diseases through increased availability of natural histories.In closing, Dr. Woodcock used remarkably similar language as Dr. Shuren in stating that FDA’s base funding is not adequate to achieve these objectives. CDER has done a lot of the preliminary legwork already, but CDER will not see fruits of this work until it has the additional funding necessary to implement the above transformational priorities.Editorial note: The Analysis and Commentary section is written by Steven Grossman, Deputy Executive Director of the Alliance for a Stronger FDA.